NextCell Pharma AB: NextCell cell therapy shows long-term effect in two independent diabetes studies "ProTrans-Repeat" and "ProTrans-Obs" - live presentation
- Thursday, October 27, CET 3:00 pm
Below follows an update on the Company's completed and ongoing clinical trials with the drug candidate ProTrans, which has been developed for type-1 diabetes, autoimmune and inflammatory diseases.
ProTrans-1. A 2019 completed open-label dose-escalation Phase-1 study in nine type-1 diabetes patients demonstrated a) good safety and b) dose-dependent efficacy regarding preservation of endogenous insulin production one year after treatment.
ProTrans-2. A since 2020 completed placebo-controlled Phase-2 study in 15 type-1 diabetes patients showed significant treatment efficacy (p<0.05), in which ProTrans-treated patients maintained 90% of their endogenous insulin production one year after treatment, versus 53% in the placebo group.
ProTrans-Repeat is an open-label follow-up study in the patients who participated in ProTrans-1 above. The nine patients were treated, after first treatment, with an additional equal dose they had received about one-two years earlier in ProTrans-1. On October 18 this year, an interim analysis after the second treatment was presented, which concludes that treatment with the highest dose gives significant the best long-term effect regarding the preservation of one's own insulin production. On average, the three cohorts retained low-, medium-, and high-dose, 41%, 45%, and 81% of their insulin-producing ability at the first time of treatment after about 3.5-4 years. The long-term effect is followed up for another two years.
ProTrans-Obs (11 patients, ongoing). Long-term follow-up of patients who previously participated in ProTrans-2. The study lasts for 5 years and 3-year interim results are presented here: " out of 14 patients who completed ProTrans-2, 6 ProTrans-treated and 5 placebo treated patients accepted to participate in follow-up study n ProTrans-Note after completion of the study, where they are followed for another 5 years. In the study, the patients' endogenous insulin production is measured semi-annually and a 3-year follow-up has been done. The results of this interim analysis show statistically significant treatment effect at all analyzed time points (p<0.05).
ProTrans-Young (66 patients, ongoing) is a Phase 1/2 study where the first part is an open-label safety study with 6 children newly diagnosed with type-1 diabetes. All patients have been treated and a safety committee is expected to recommend continuation with Part 2 in early November.
The second part of the study is a randomized, placebo-controlled double-blinded study with 30 patients aged 12-21 years and 30 patients aged 7-11 years.
Pediatric Investigation Plan, PIP. A pediatric development plan has been submitted to the European Medicines Agency's Expert Committee on Drug Development for the Treatment of Children. The plan describes the clinical trial program until commercialization and is a requirement for phase-3 studies. The application includes ProTrans-3, a pivotal phase III study for including both adults and children that, in the event of a positive outcome, can give ProTrans marketing approval.
Protrans19+SE (9 patients, ongoing). Swedish open-label dose escalation study (phase 1) with ProTrans for the treatment of adult patients (not type-1 diabetes), who suffered from severe pneumonia as a result of Sars-CoV-2 infection.
Protrans19+CA (48 patients, ongoing). Canadian randomized, placebo-controlled, double-blinded (Phase 2) study for the treatment of adult patients (not diabetes), who suffered from severe pneumonia as a result of Sars-CoV-2 infection.